News & Stories
See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.
DTx Pharma Achieves Second Milestone in Study to Advance Treatments for CMT1A
The CMT Research Foundation is pleased to announce that our partner DTx Pharma just completed the second milestone in a study we’re funding which uses their proprietary technology to suppress the overexpression of PMP22, the protein that causes CMT1A. Their work has...
Nothing Stops Innovation: Results from BIO 2020
By: Susan Ruediger, CEO, CMT Research Foundation “We’re not currently working in CMT, but we know we should be.” “We think our drug could work for CMT, but it has never been tested.” “CMT is definitely on our interest list for expansion, but we need to learn more...
CMT Genetic Testing: What’s Involved?
During the past several decades, researchers have discovered more than 100 genetic mutations that cause CMT. In the same time frame, scientists have developed tests that can look at parts of someone’s genetic makeup and identify changes that could cause certain...
Understanding CMT Genetics
CMT is a genetic disorder, meaning it’s caused by a change, or mutation, in your genes. If you have CMT, you might be wondering how you got it. It’s likely that other people in your family have it, too — though this isn’t always the case. To understand how and why,...
New Research Project to Advance Treatments for CMT1B
The CMT Research Foundation is pleased to announce a new partnership with The Jackson Laboratory that will help us better understand CMT1B and potentially lead to the discovery of treatments. CMT1B is the third most common form of CMT. It’s caused by a defect in the...
DTx Pharma Milestone 1 Completed
Our partners at DTx Pharma continue to make great progress on suppressing the overexpression of PMP22, which causes CMT1A. DTx Pharma’s proprietary technology has the potential to render RNA-based therapeutics such as ASO and siRNA more potent, safer and longer-acting...
Gene Therapy for Axonal Neuropathies
There are efforts underway to develop gene therapies for several of the CMT types 2 and 4.
Milestone 2 Completed for CMT1A Project from Cyprus Institute of Neurology and Genetics
In February 2020, Dr. Kleopa completed milestone 2 of the 24-month project and the results continue to be encouraging. The goal during this 6-12 month period of the project was to further optimize the amount of PMP22 gene silencing in a mouse model of CMT1A. Dr....
Creating multiple opportunities for success in CMT 1A
By Grace Pavlath, PhD By now you’ve likely heard that CMTRF is funding three genetic-based projects focused on therapy development for CMT1A: DTx Pharma, Shift Pharma and Cyprus Institute of Neurology and Genetics. Funding multiple complementary approaches gives...
CMT Research Foundation Funds Shift Pharmaceuticals to Advance Search for CMT1A Treatment
Shift to Develop and Analyze Novel Series of Drugs Designed to Control Expression of PMP22 Gene The CMT Research Foundation (CMTRF), a nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, today announced it has partnered with...
Important Research News Announcements
- Genomic Studies in Charcot-Marie-Tooth Disease July 31, 2024
- Charcot-Marie-tooth disease type 2A: An update on pathogenesis and therapeutic perspectives March 5, 2024
- Accelerate Clinical Trials in CMT (ACTCMT) Study March 1, 2024
- Creation of a Schwann Cell Gene Regulatory Network February 29, 2024
- Activation of XBP1s attenuates disease severity in models of proteotoxic Charcot-Marie-Tooth type 1B [Preprint] February 2, 2024
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